CHICAGO – Esya Labs, which is developing several ground-breaking novel research assays, today has announced that Novartis is among the first companies to use its technology. Novartis, a global healthcare company, has used a patented assay developed by Esya Labs to accelerate drug discovery efforts for Mucolipidosis Type IV, a rare inherited metabolic...
Latest News
Zurich, Switzerland – Glioblastoma is a particularly aggressive brain tumour that at present is incurable. Cancer doctors can extend patients’ life expectancy through operations, radiation, chemotherapy or surgical interventions. Nevertheless, half of patients die within twelve months of diagnosis. Drugs that are effective against brain tumours are difficult to find,...
DEER PARK, Ill. — Eton Pharmaceuticals (Nasdaq: ETON), an innovative pharmaceutical company focused on developing, acquiring, and commercializing products to address unmet needs in patients suffering from rare diseases, today announced the commercial availability of Nitisinone Capsules for the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with dietary...
Brussels, Belgium – Multiple sclerosis (MS), an inflammatory disease of the central nervous system, affects more than 1 million people in Europe and an estimated 2.8 million worldwide. MS is characterized by an inflammation-related loss and damage of the myelin layer in the brain and spinal cord, which insulates the...
A group of more than 40 EU lawmakers backed the idea of an EU action plan for rare diseases, in a letter addressed to European Commission President Ursula von der Leyen.
EMERYVILLE, Calif. – Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat solid tumors, today announced the initiation of ARYA-2, a Phase I/II open-label, dose escalation clinical trial of ET140203 ARTEMIS® T-cell therapy in pediatric patients with relapsed or refractory hepatoblastoma (HB), hepatocellular neoplasm not otherwise specified (HCN-NOS),...
EMERYVILLE, Calif. – Eureka Therapeutics, Inc. announced today that Eureka and Memorial Sloan Kettering Cancer Center (MSK) entered into a license agreement with Sanofi for the non-CAR use of a novel, human binding domain targeting GPRC5D (G Protein-Coupled Receptor Family C Group 5 Member D). The GPRC5D binding domain was discovered using...
LONDON, UK – Vertex Pharmaceuticals Inc. (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD)...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one...
NEW YORK, NY — Pfizer Inc. (NYSE: PFE) announced today that the European Commission (EC) has granted marketing authorization for VELSIPITY® (etrasimod) in the European Union to treat patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response,...